(WFRF:(Bishop J. A.)) srt2:(2015-2019)
Search: (WFRF:(Bishop J. A.)) srt2:(2015-2019) > (2018) > Nusinersen versus S...
- 9 of 16
- Previous record
- Next record
- To hitlist
| Fältnamn | Indikatorer | Metadata |
|---|---|---|
| 000 | 04575naa a2200649 4500 | |
| 001 | oai:gup.ub.gu.se/265106 | |
| 003 | SwePub | |
| 008 | 240528s2018 | |||||||||||000 ||eng| | |
| 024 | 7 | a https://gup.ub.gu.se/publication/2651062 URI |
| 024 | 7 | a https://doi.org/10.1056/NEJMoa17105042 DOI |
| 040 | a (SwePub)gu | |
| 041 | a eng | |
| 042 | 9 SwePub | |
| 072 | 7 | a ref2 swepub-contenttype |
| 072 | 7 | a art2 swepub-publicationtype |
| 100 | 1 | a Mercuri, E.4 aut |
| 245 | 1 0 | a Nusinersen versus Sham Control in Later-Onset Spinal Muscular Atrophy |
| 264 | 1 | c 2018 |
| 520 | a BACKGROUND Nusinersen is an antisense oligonucleotide drug that modulates pre-messenger RNA splicing of the survival motor neuron 2 (SMN2) gene. It has been developed for the treatment of spinal muscular atrophy (SMA). METHODS We conducted a multicenter, double-blind, sham-controlled, phase 3 trial of nusinersen in 126 children with SMA who had symptom onset after 6 months of age. The children were randomly assigned, in a 2: 1 ratio, to undergo intrathecal administration of nusinersen at a dose of 12 mg (nusinersen group) or a sham procedure (control group) on days 1, 29, 85, and 274. The primary end point was the least-squares mean change from baseline in the Hammersmith Functional Motor Scale-Expanded (HFMSE) score at 15 months of treatment; HFMSE scores range from 0 to 66, with higher scores indicating better motor function. Secondary end points included the percentage of children with a clinically meaningful increase from baseline in the HFMSE score (>= 3 points), an outcome that indicates improvement in at least two motor skills. RESULTS In the prespecified interim analysis, there was a least-squares mean increase from baseline to month 15 in the HFMSE score in the nusinersen group (by 4.0 points) and a least-squares mean decrease in the control group (by -1.9 points), with a significant between-group difference favoring nusinersen (least-squares mean difference in change, 5.9 points; 95% confidence interval, 3.7 to 8.1; P< 0.001). This result prompted early termination of the trial. Results of the final analysis were consistent with results of the interim analysis. In the final analysis, 57% of the children in the nusinersen group as compared with 26% in the control group had an increase from baseline to month 15 in the HFMSE score of at least 3 points (P< 0.001), and the overall incidence of adverse events was similar in the nusinersen group and the control group (93% and 100%, respectively). CONCLUSIONS Among children with later-onset SMA, those who received nusinersen had significant and clinically meaningful improvement in motor function as compared with those in the control group. (Funded by Biogen and Ionis Pharmaceuticals; CHERISH ClinicalTrials. gov number, NCT02292537.) | |
| 650 | 7 | a MEDICIN OCH HÄLSOVETENSKAPx Klinisk medicinx Pediatrik0 (SwePub)302212 hsv//swe |
| 650 | 7 | a MEDICAL AND HEALTH SCIENCESx Clinical Medicinex Pediatrics0 (SwePub)302212 hsv//eng |
| 653 | a functional motor scale | |
| 653 | a lumbar puncture | |
| 653 | a headache | |
| 653 | a children | |
| 653 | a severity | |
| 653 | a backache | |
| 653 | a protein | |
| 653 | a trials | |
| 653 | a General & Internal Medicine | |
| 700 | 1 | a Darras, B. T.4 aut |
| 700 | 1 | a Chiriboga, C. A.4 aut |
| 700 | 1 | a Day, J. W.4 aut |
| 700 | 1 | a Campbell, C.4 aut |
| 700 | 1 | a Connolly, A. M.4 aut |
| 700 | 1 | a Iannaccone, S. T.4 aut |
| 700 | 1 | a Kirschner, J.4 aut |
| 700 | 1 | a Kuntz, N. L.4 aut |
| 700 | 1 | a Saito, K.4 aut |
| 700 | 1 | a Shieh, P. B.4 aut |
| 700 | 1 | a Tulinius, Mar,d 1953u Gothenburg University,Göteborgs universitet,Institutionen för kliniska vetenskaper, Avdelningen för pediatrik,Institute of Clinical Sciences, Department of Pediatrics4 aut0 (Swepub:gu)xtulim |
| 700 | 1 | a Mazzone, E. S.4 aut |
| 700 | 1 | a Montes, J.4 aut |
| 700 | 1 | a Bishop, K. M.4 aut |
| 700 | 1 | a Yang, Q.4 aut |
| 700 | 1 | a Foster, R.4 aut |
| 700 | 1 | a Gheuens, S.4 aut |
| 700 | 1 | a Bennett, C. F.4 aut |
| 700 | 1 | a Farwell, W.4 aut |
| 700 | 1 | a Schneider, E.4 aut |
| 700 | 1 | a De Vivo, D. C.4 aut |
| 700 | 1 | a Finkel, R. S.4 aut |
| 700 | 1 | a Cherish Study Grp, Cherish Study Grp4 aut |
| 710 | 2 | a Göteborgs universitetb Institutionen för kliniska vetenskaper, Avdelningen för pediatrik4 org |
| 773 | 0 | t New England Journal of Medicineg 378:7, s. 625-635q 378:7<625-635x 0028-4793 |
| 856 | 4 8 | u https://gup.ub.gu.se/publication/265106 |
| 856 | 4 8 | u https://doi.org/10.1056/NEJMoa1710504 |
Find in a library
- New England Journal of Medicine (Search for host publication in LIBRIS)
To the university's database
- 9 of 16
- Previous record
- Next record
- To hitlist
Find more in SwePub
- By the author/editor
- Mercuri, E.
- Darras, B. T.
- Chiriboga, C. A.
- Day, J. W.
- Campbell, C.
- Connolly, A. M.
- show more...
- Iannaccone, S. T ...
- Kirschner, J.
- Kuntz, N. L.
- Saito, K.
- Shieh, P. B.
- Tulinius, Mar, 1 ...
- Mazzone, E. S.
- Montes, J.
- Bishop, K. M.
- Yang, Q.
- Foster, R.
- Gheuens, S.
- Bennett, C. F.
- Farwell, W.
- Schneider, E.
- De Vivo, D. C.
- Finkel, R. S.
- Cherish Study Gr ...
- show less...
- About the subject
-
- MEDICAL AND HEALTH SCIENCES
- MEDICAL AND HEAL ...
- and Clinical Medicin ...
- and Pediatrics
- Articles in the publication
- New England Jour ...
- By the university
- University of Gothenburg
Search outside SwePub
- Extend your search to:
- Google Book Search
- Google Scholar
Kungliga biblioteket hanterar dina personuppgifter i enlighet med EU:s dataskyddsförordning (2018), GDPR. Läs mer om hur det funkar här.
Så här hanterar KB dina uppgifter vid användning av denna tjänst.
- Copyright © LIBRIS - National Library Systems
- LIBRIS.kb.se
